University of Minnesota research team discovers new therapy potential for Duchenne Muscular Dystrophy
Approach is about 150,000 times more effective than similar existing treatments
MINNEAPOLIS/ST. PAUL (10/21/2025) — Researchers in the University of Minnesota College of Science and Engineering and Medical School have identified a new approach that could change the way Duchenne Muscular Dystrophy (DMD) is treated.
The findings were recently published in PNAS, one of the world's most-cited and comprehensive multidisciplinary scientific journals.
DMD is a genetic disease that causes muscles to weaken and deteriorate over time, often leading to severe disability and early death. Globally, about 20,000 children are diagnosed with DMD each year. Currently, there is no cure.
The researchers developed a new type of therapy using a “bottlebrush polymer,” a highly branched synthetic molecule designed to protect muscle cell membranes from damage. This approach directly targets fragile muscle membranes that break down under stress, which is the root problem in DMD.
“This work, made possible by a cross-disciplinary collaboration, establishes a new class of synthetic molecules for muscle membrane protection with potential to spark a revolution in novel therapeutics for muscular dystrophy,” said the study's corresponding author Joe Metzger, Ph.D., a Maurice B. Visscher Endowed Land-Grant chair in physiology, and professor and chair of the Department of Integrative Biology and Physiology at the University of Minnesota Medical School. “We hope this work will illuminate the ultimate pathway toward effective clinical treatment for DMD patients.”
In this study, the bottlebrush polymer was shown to be about 150,000 times more effective than existing polymer treatments at restoring muscle function. When tested in preclinical models of DMD, the therapy prevented muscle damage in the limbs and diaphragm, and even protected the heart from stress-related injury and death.
Because DMD begins in early childhood, researchers believe that, in conjunction with newborn screening for DMD, this therapy could potentially preserve muscle strength and extend quality of life. The therapy may also hold promise for other inherited or acquired diseases that involve muscle damage.
"This translational work combined innovative macromolecular engineering with insightful physiological experiments, bridging the College of Science and Engineering and the Medical School in an inspirational interdisciplinary collaboration," said co-author of the study Frank Bates, Ph.D., a regents professor in the University of Minnesota Department of Chemical Engineering and Materials Science in the University of Minnesota College of Science and Engineering.
Future work will focus on securing funding to launch clinical trials.
The study was supported by the National Institutes of Health, the Muscular Dystrophy Foundation, University of Minnesota Marzolf Foundation, the American Heart Association and the University of Minnesota Medical School.